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437 Lipid droplet accumulation was observed in brown adipose tissue and heart in a mouse model of Friedreichs ataxia, as well as in Frataxin-deficient rat cardiomyocytes and IPSC-derived cardiomyocytes

However, it is important to note that these observations come exclusively from animal studies and that safety data in humans remain very limited

The facilities being built and qualified now are designed for continuous manufacturing, kilogram-scale purification, and the analytical complexity of modified peptides with multiple critical quality attributes requiring real-time monitoring